How Did Transthyretin Amyloid Cardiomyopathy Progress in Patients Who Took Placebo in the Study ATTR-ACT? A Plain Language Summary | oneAMYLOIDOSISvoice
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How Did Transthyretin Amyloid Cardiomyopathy Progress in Patients Who Took Placebo in the Study ATTR-ACT? A Plain Language Summary

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source: Future Cardiology

year: 2022

authors: Jose Nativi-Nicolau, Daniel P. Judge, James E. Hoffman, Balarama Gundapaneni, Denis Keohane, Marla B. Sultan, Martha Grogan

summary/abstract:

What Is This Plain Language Summary About?
This plain language summary describes some results of a study called ATTR-ACT. This was the first large study to include people with wild-type and hereditary transthyretin amyloid cardiomyopathy (ATTR-CM for short). ATTR-CM is a type of heart disease that happens when abnormal clumps of protein build up in the heart. This build-up prevents the heart from working properly, causing a condition called heart failure. Wild-type ATTR-CM happens for unknown reasons in some people as they get older. Hereditary ATTR-CM can happen because of changes in people’s genes (known as gene variants or mutations).
 
Important Information About ATTR-ACT
In this study, 441 people with ATTR-CM took either a medicine called tafamidis or a placebo (a capsule that looked like tafamidis but didn’t contain any active medicine) by mouth for 30 months, once a day. The researchers’ main aim was to find out how tafamidis treatment affected the risk of people dying or being admitted to the hospital for heart problems. They found that tafamidis lowered these risks by about one-third compared with placebo.
 
What Else Did Researchers Find Out in ATTR-ACT?
As described in this summary, after ATTR-ACT was completed, researchers looked back at the results from people who took placebo to learn how ATTR-CM progressed without treatment. The researchers found that about 4 in 10 people with wild-type ATTR-CM who took placebo died and 6 in 10 were admitted to the hospital because of heart problems over 30 months. People who took placebo also could not walk as far at the end of the study as they did at the start because their heart function worsened over time.
 
Why Are These Results Important?
By showing how ATTR-CM affects people’s health when they do not take treatment, these results highlight the benefits of early diagnosis and treatment of ATTR-CM.
 
Where Can I Find More Information on the Study?
This summary only contains information from the full scientific article, which is published in the journal ESC Heart Failure and is called ‘Natural history and progression of transthyretin amyloid cardiomyopathy: insights from ATTR-ACT’. This article is available to readers at no cost and can be read at: https://www.oneamyloidosisvoice.com/rcuratenew/natural-history-progression-transthyretin-amyloid-cardiomyopathy-insights-attr-act/
 
organization: Mayo Clinic, Jacksonville, USA; Medical University of South Carolina, USA; University of Miami, USA; Pfizer Inc, Groton, USA; Pfizer Inc, New York, USA; Mayo Clinic, Rochester, USA

DOI: 10.2217/fca-2021-0150

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